Read our patient story from Feike.
Welcome to IamGSD!
Our information is prepared in English.
On the “Languages” button you can learn about using the Google Chrome browser to enable you to read our automatically translated site in your choice of 100 languages.
Many of our printed publications are available in several languages in addition to English.
MUSCLE GLYCOGEN STORAGE DISEASE
GLOBAL WALKING CHALLENGE
MAY 21st to 27th
An incentive to get moving, with our 9th Global Walking Challenge. Join us in this challenge to walk the length of long distance trails around the world! You join in virtually, walking wherever you are, and sending in your mileage each day. Send us comments and photos as well! We will add the miles all together and tick off some amazing trails. The big challenge is to culminate with finishing the California Coastal Trail at 1931 km (1199 miles).
RARE DISEASE DAY
Help to promote Rare Disease Day, a major international effort to raise awareness of the 7,000 or so rare diseases which affect around 300 million people worldwide. It is held on the last day of February each year. Visit their website for ideas on how you can participate. In particular, this year you can share your story of your rare disease by submitting a brief account via their website.
UPDATE ON REN001 PHASE 1B TRIAL
A Reneo Pharmaceuticals, Inc. update included details on their trial in patients with McArdle disease. This showed an adequate safety profile with no serious adverse events. After 12 weeks of treatment, participants showed an average increase of 38.5% in fat oxidation compared to baseline. However, the increase in fat oxidation did not result in major changes in the distance walked during the 12-minute walk test and no corresponding changes in heart rate, fatigue or pain were noted. Reneo does not plan to pursue a Phase 2 study. They will continue to partner with us to further our advocacy and education mission.
CLINICAL PRACTICE GUIDELINES PUBLISHED
A huge landmark in the progress of the understanding and treatment of GSD5 & 7 has been passed. The Clinical Practice Guidelines for these conditions, prepared by an international team of 18 clinicians, researchers and patient advocates led by IamGSD, has been published in Neuromuscular Disorders, the official journal of the World Muscle Society December issue. The paper and Supplementary Material are available free under Open Access.
IAMGSD’S THOUGHTS ARE WITH UKRAINE
Since Russia launched a military invasion of Ukraine on February 24, fighting has caused civilian casualties and pushed over 1m Ukrainians to flee to neighbouring countries. We are particularly concerned for those living with muscle GSDs and the disruptions in healthcare services they are facing. We are also concerned about the physical difficulties these individuals will face as they flee from the war. IamGSD stands in solidarity with the people of Ukraine and with the international community as they continue to support all those affected.
TRAINING SUPPORT PAGE LAUNCHED
Our new page for Training Support is now made public in its final draft form. The page has the new “At the gym” leaflet and other resources intended to support physical therapists and personal trainers who are assisting people with McArdle’s. Feedback is requested by January 31 in order to assist us to complete the final versions of these resources.
IAMGSD promotes walking courses to help people improve their condition. These have so far been held in the USA, UK and Germany, with more countries to be included in future.